RecruitingRare disease

A study of guanabenz acetate for children with vanishing white matter disease who started treatment in early childhood

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study involves patients with vanishing white matter, a rare inherited brain disease that affects the white matter of the brain, which is the part responsible for sending signals between different brain areas. White matter gradually disappears in this condition, leading to problems with movement, balance, and other body functions. The study will use guanabenz acetate, a medicine given as capsules taken by mouth. Different doses of guanabenz capsules will be used during the study.

The purpose of this study is to evaluate the long-term effects of guanabenz in patients with early childhood onset vanishing white matter by comparing their progress to information from similar patients who were not treated with this medicine in the past. The study will look at whether guanabenz helps maintain or improve the ability to walk, overall survival, quality of life, and various abilities related to movement, communication, and daily activities. The study will also examine changes in the brain using special imaging techniques and will check how the body processes the medicine by measuring drug levels in the blood.

This is an extension study, meaning it continues treatment for patients who already participated in a previous study called VWM1. Patients will continue taking guanabenz for several more years, and their progress will be carefully monitored through regular visits and assessments. During the study, doctors will check for any unwanted effects of the medicine and measure various aspects of movement ability, thinking skills, and overall functioning using standardized tests and questionnaires. Brain scans using MRI will be performed to see how the white matter changes over time with treatment.

The research process

The trial runs in 9 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Continuation from previous study
This study is an extension of the previous VWM1 study. Your child will continue treatment with guanabenz after completing the VWM1 study.
The medication will be taken by mouth in the form of capsules.
Step 2
Treatment with guanabenz
Your child will receive guanabenz acetate in capsule form. The capsules are available in different strengths: 1 mg, 2 mg, 4 mg, 8 mg, 16 mg, and 24 mg.
The specific dosage and frequency of administration will be determined based on your child's individual needs.
The medication is taken orally, meaning it is swallowed by mouth.
Step 3
Regular study visits and assessments
Your child will attend scheduled assessments throughout the study. These visits may take place at the study site or through video consultation, depending on what is specified in the study plan.
During these visits, various evaluations will be performed to monitor your child's condition and response to treatment.
Step 4
Physical function assessments
Your child's ability to walk will be assessed. This includes checking whether your child can walk at least 10 steps without support or with light support of one hand.
Several standardized assessments will be used to measure motor function, which refers to your child's ability to move and perform physical activities. These include the Gross Motor Function Measure (GMFM 88), which evaluates movement abilities.
Additional assessments will measure your child's manual ability (how well your child uses their hands), communication function, and eating and drinking abilities.
Step 5
Quality of life and daily functioning evaluations
Your child's quality of life and level of disability will be evaluated using various questionnaires and scales.
These assessments will measure how your child functions in daily activities and overall well-being.
If your child is 8 years old or older, both you and your child may be asked to complete questionnaires about your child's health and quality of life.
Step 6
Brain imaging scans
Your child will undergo brain MRI scans. MRI stands for Magnetic Resonance Imaging, which is a type of scan that creates detailed pictures of the brain.
These scans will use specialized techniques to examine the white matter in the brain and measure changes over time.
The imaging will include different methods such as Diffusion Tensor Imaging, Chemical Shift Imaging, Neurite Orientation Dispersion and Density Imaging, and Myelin Water Fraction Imaging. These are advanced techniques to assess the condition of brain tissue.
Step 7
Blood sample collection
Blood samples will be collected from your child to measure the levels of guanabenz in the blood.
These measurements will help determine how the medication is processed by your child's body, including the highest concentration reached, how long the medication stays in the body, and the expected lowest level between doses.
Step 8
Safety monitoring
Throughout the study, your child will be monitored for any adverse events, which are unwanted or harmful effects that may occur during treatment.
The frequency, severity, and impact of any adverse events on your child's daily life will be recorded and assessed.
Step 9
Study duration
The study is expected to run from December 2025 until November 2029.
Your child will continue in the study for the duration specified by the study plan, with regular follow-up throughout this period.

Who can join the trial?

6 criteria

The patient's parents or legal guardians (people who have legal responsibility for the child) must sign a document called an informed consent form, which shows they understand what the study is about and what will be required
The parents or legal guardians must be willing to have their child take part in the study and attend all scheduled visits, either at the study location or through video calls as the study requires
The parents or legal guardians must be willing and able to follow all study rules and procedures, including staying in contact with the study team for the entire length of the study
The parents or legal guardians must be able to follow the restrictions and rules described in the study plan
The patient must have finished a previous study called VWM1 study
The patient must live close enough to Amsterdam to be able to travel there when needed

Who cannot join the trial?

1 criterion

No exclusion criteria have been specified in the available study information

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Verified sites

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Amsterdam UMC
Verified
Amsterdam, The Netherlands

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Investigated drugs

Guanabenz is a medication being tested in this study for patients with vanishing white matter disease. This medicine is being investigated to see if it can help treat this condition over a long period of time and to check if it is safe for patients to use.

Guanabenz Acetate

What is already known about the treatment

Guanabenz – Guanabenz is a medication that is being investigated in clinical trials for the treatment of vanishing white matter disease, a rare genetic brain disorder that primarily affects children. This drug is currently in the research phase for this specific condition, though it has been previously approved for other medical uses. Guanabenz works at the molecular level by helping to reduce stress on cells and potentially protecting the brain's white matter from deterioration. In this clinical trial, researchers are studying whether long-term treatment with guanabenz can slow down or stop the progression of symptoms in young children who develop this disease before the age of six years.

Investigated diseases

Vanishing White Matter Disease – Vanishing white matter disease is a rare inherited disorder that affects the brain's white matter, which is the tissue responsible for transmitting signals between different parts of the brain. The disease causes the white matter to gradually break down and disappear, being replaced by fluid-filled spaces. It primarily affects young children, though onset can occur at various ages. The condition leads to progressive loss of motor skills, including the ability to walk and maintain balance. Episodes of rapid deterioration often occur following fever, minor head trauma, or periods of severe fright. Over time, affected individuals experience increasing difficulties with movement, coordination, and other neurological functions.